Drug intelligence / Profile preview

APR-OM33

Development stage
Preclinical
Lead developer
APR Applied Pharma Research
Modality
Small Molecules
Administration
Oral
01

Overview

APR-OM33 is an investigational amino acid-based formulation developed by APR Applied Pharma Research (a subsidiary of Relief Therapeutics) for the treatment of Maple Syrup Urine Disease (MSUD). It is built upon the proprietary Organic Acid Technology (OAT) platform, which is designed to modulate the release of amino acids to mimic the slow, continuous absorption of natural dietary proteins. MSUD is a rare metabolic disorder characterized by the body's inability to process branched-chain amino acids (BCAAs), specifically leucine, isoleucine, and valine. By providing a BCAA-free protein substitute with a controlled-release profile, APR-OM33 aims to prevent the rapid spikes in blood leucine levels that are toxic to the central nervous system, thereby improving metabolic control and long-term outcomes for patients.

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